Experimental Pill Breakthrough Sees Cancer Vanish in a Third of Leukaemia Patients

Calendar March 23, 2023 | Posted by admin

Terminal leukaemia patients who were not responding to treatment now have hope for a cure, thanks to a new experimental pill called revumenib. This drug has completely eliminated cancer in a third of the participants in a long-awaited clinical trial in the United States. Although not all patients showed complete remission, scientists remain hopeful as the results indicate that the pill might pave the way to a cure for leukaemia in the future.

“We’re incredibly hopeful by these results of patients that received this drug. This was their last chance,” said study co-author Dr Ghayas Issa, a leukaemia physician at the MD Anderson Cancer Center at the University of Texas. “They have progressed on multiple lines of therapy and a fraction of them, about half, had disappearance of their leukaemia cells from their bone marrow,” he told Euronews Next.

Acute myeloid leukaemia (AML) is a type of cancer that attacks the bone marrow, where blood cells are produced, and causes the uncontrolled production of defective cells. Revumenib is a new class of targeted therapy for acute leukaemia that inhibits a specific protein called menin. The drug works by reprogramming leukaemia cells back into normal cells. Menin is involved in the complex machinery that gets hijacked by leukaemia cells and causes normal blood cells to turn into cancerous ones. By using revumenib, Issa explained, the engine is turned off and leukaemia cells are turned back into normal cells, resulting in remission.

This formula has already saved 18 lives as part of the clinical trial, whose promising results were published this month in Nature. The preliminary results showed that 53 per cent of patients responded to revumenib, and 30 per cent had a complete remission with no cancer detectable in their blood. Based on the data from this trial, in December 2022 the US Food and Drug Administration granted revumenib “breakthrough therapy designation” to help fast-track its development and regulatory review.

“This is definitely a breakthrough and it’s a result of years of science. A lot of groups had worked hard in the lab to understand what is causing these leukaemias,” Issa said. However, he explained that the drug does not work for all patients. It is for a specific subset of leukaemias that generally have missing or mislabeled genes or a chromosome fusion. The experimental pill targets the most common mutation in acute myeloid leukaemia, a gene called NPM1, and a less common fusion called KMT2A. Combined, these mutations are estimated to occur in about 30 to 40 per cent of people with acute myeloid leukaemia.

The phase 1 trial enrolled 68 patients at nine US hospitals. All of them had seen their leukaemia return after other treatments or had never responded well to traditional chemotherapy drugs in the first place.

Source: https://www.euronews.com/next/

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Scientists Have Possibly Cured HIV

Calendar February 16, 2022 | Posted by admin

An American research team reported that it has possibly cured HIV in a woman for the first time. Building on past successes, as well as failures, in the HIV-cure research field, these scientists used a cutting-edge stem cell transplant method that they expect will expand the pool of people who could receive similar treatment to several dozen annually.

Their patient stepped into a rarified club that includes three men whom scientists have cured, or very likely cured, of HIV. Researchers also know of two women whose own immune systems have, quite extraordinarily, apparently vanquished the virus. Carl Dieffenbach, director of the Division of AIDS at the National Institute of Allergy and Infectious Diseases, one of multiple divisions of the National Institutes of Health that funds the research network behind the new case study, told NBC News that the accumulation of repeated apparent triumphs in curing HIV “continues to provide hope.”

“It’s important that there continues to be success along this line,” he said.

In the first case of what was ultimately deemed a successful HIV cure, investigators treated the American Timothy Ray Brown for acute myeloid leukemia, or AML. He received a stem cell transplant from a donor who had a rare genetic abnormality that grants the immune cells that HIV targets natural resistance to the virus. The strategy in Brown’s case, which was first made public in 2008, has since apparently cured HIV in two other people. But it has also failed in a string of others. This therapeutic process is meant to replace an individual’s immune system with another person’s, treating their cancer while also curing their HIV. First, physicians must destroy the original immune system with chemotherapy and sometimes irradiation. The hope is that this also destroys as many immune cells as possible that still quietly harbor HIV despite effective antiretroviral treatment. Then, provided the transplanted HIV-resistant stem cells engraft properly, new viral copies that might emerge from any remaining infected cells will be unable to infect any other immune cells.

Source: https://www.nbcnews.com/

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